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Isis and Roche form an alliance to combat Huntington's
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Huntington’s disease is an inherited genetic disorder affecting the brain that leads to progressive loss of both mental and physical faculty, motor control and psychiatric problems. It is an autosomal dominant mutation of either of the copies of the gene called huntingtin, with heritability of 50%, meaning that a child with a parent suffering from Huntington’s disease has a 50% chance to develop it. It usually presents symptoms between the ages of 35 and 50, but can present itself in any point in life, ranging from childhood to old age. Once diagnosed, it continues to progress leading to a steady decline in the individuals health, ultimately resulting in death of the weekend organism usually due to pneumonia, emphysema or similar condition. So far, there have been only a few ways to ease the symptoms and none to combat the disease itself.

April, 2013, two pharmaceutical giants, Isis Pharmaceuticals and Roche Pharmaceuticals have pooled their recourses in an alliance to attempt and cure Huntington’s disease combining Isis’ antisense oligonucleotide technology and Roches expertise in developing neurodegenerative medicine. The collaboration will include a combination of Isis’ antisense oligonucleotide technology and Roshe’s new “brain shuttle” program, to attempt and increase the antisense oligonucletoide penetration into the brain by adding systematic administration to Isis’ technology.
At first, the research will focus on Isis’ drug candidate for the blockage of all forms of the huntingtin protein (the main product of the huntingtin gene, a malfunctioning protein which is the main causative of the disease) production, hoping it will show effective in halting the degeneration caused by Huntington’s, or even prevent it altogether. Isis is also conducting research into experimental treatments that specifically block the production of the disease-causing forms of the huntingtin protein, which has the potential to treat subsets of Huntington’s patients. In a parallel research, Roche’s scientists will combine their brain shuttle technology to allow the systematic administration of the antisense oligonucleotide drug directly into the brain of asymptomatic patients.

Under the terms of the agreement, Roche will make a payment of $30 million to Isis, with total payments related to license fee and pre- and post-licensing milestone payments reaching potentially $362 million, including a further $80 million in potential commercial milestone payments. In addition, Isis will receive royalties on the sales of the drugs. Roche has the option to license the drugs from Isis through the completion of the first Phase I trial and additional unstated payments.

By this agreement, Isis is responsible for the development and testing of the antisense drug, and Roche will provide it’s brain shuttle technology to collaborate on the developmental stages of the drug. Roche will be responsible for the marketing, logistics, production and distribution of the drug.

“Huntington's is a severely debilitating neurodegenerative disease and a large unmet medical need. Patients experience gradually worsening motor function and psychological disturbances, with a significant shortening of life expectancy after the disease is diagnosed. Treatments are urgently needed, and we believe that the Isis approach in combination with Roche's brain shuttle represent one of the most advanced programs targeting the cause of HD with the aim of slowing down or halting the progression of this disease.” said Luca Santarelli, head of neuroscience and small molecules research at Roche.

B. Lynne Parshall, COO of Isis said: “We believe that Roche's expertise in developing CNS drugs, along with their clinical development experience, will greatly enhance our development efforts for this program and allow us to move forward more rapidly. In addition, by utilizing Roche's brain shuttle technology with our antisense drug discovery capabilities, we have the potential to significantly improve the therapeutic potential for this program,”

“By partnering our more complex and nuanced research and development programs earlier in development, like our Huntington's disease CNS program, we add value and resources with partners that bring unique benefits.”
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Isis and Roche form an alliance to combat Huntington's00